Sign on to protect the Orphan Drug Tax Credit

Stand with NORD and the entire Rare Disease Community to urge congress to reconsider the proposal to repeal the Orphan Drug Tax Credit.

“Repeal of this provision would squelch medical research and innovation. Repeal would remove one of the major incentives to finding cures and treatments for some of the most challenging diseases”, says Peter Saltonstall, President and CEO, National Organization for Rare Disorders (NORD).

Drug companies need more incentives to develop rare disease treatments, not less.  Congress must hear from the patient community that the Orphan Drug Tax Credit is important to them.

Rare disease patient organizations wishing to sign-on to NORD’s letter to Congress on this issue should write to by COB Friday March 7.

Rare Disease Day Cocktail Reception & Documentary Screening

Please join RDLA for our 4th Annual Rare Disease Cocktail Reception & Screening of the HBO Documentary Film Life According to Sam to kick-off Rare Disease Day Week in Washington, DC.

Tuesday, February 25, 2014
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Patient Orgs Sign-on to Support Congressional Letter on Accelerated Approval

In response to the FDA’s draft guidance on Expedited Programs for Serious ConditionsRepresentatives Gus Bilirakis (R-FL), GK Butterfield (D-NC), Ileana Ros-Lehtinen (R-FL), and Jim Moran (D-VA) are circulating a bipartisan letter in Congress calling on the FDA ensure the final guidance fully implements FDASIA to give rare diseases access to Accelerated Approval.  Because of the support of the patient community 24 Members of Congress have signed onto the letter.

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2nd Annual RareVoice Awards Gala

RareVoice Award nominations are open to the public.  We encourage the community to nominate individuals and organizations who advocate for legislation and policy that benefits the rare disease community.  Deadline to submit nominations is  Friday August 23, 2103.

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Sept. 20: 2013 RARE Patient Advocacy Summit


Join Global Genes Project for their 2nd Annual “RARE Patient Advocacy Summit” to be held on Friday, September 20, 2013 at The Balboa Bay Club & Resort in Newport Beach, CA. Register today!  Seating is limited for in-person participation.  Webcast registration available for those unable to attend in person.

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Oct. 7-9: US Conference on Rare Diseases & Orphan Products

US Conference on Rare Diseases & Orphan Products: The New Era in Health Care, which will be held October 7-9 in North Bethesda, MD.

This year, the third Annual Meeting of stakeholders in the rare disease/orphan product community comes at an opportune time. Two new laws – the Affordable Care Act (ACA) and the FDA Safety and Innovation Act (FDASIA) – are being implemented.

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Sign-on to Support the Smart Regulation of Health IT

Coalition Letter:  Health Information Technology Framework
Proper oversight of health IT is critical to ensuring patient safety, however such oversight must be implemented in a balanced way that also fosters innovation and encourages adoption of these technologies.  A coalition is encouraging the Administration to ensure health IT is regulated in a way that achieves the goals of improving the quality, safety and cost-effectiveness of care.

Read the letter & see the long list of supporters here:  Coalition Sign-on Letter Regarding Policy Associated with Health IT.  To add your organization to the growing list of supporters, please contact Jen Bernstein at

Urge Congress to Support $32 billion for the NIH in 2014

FASEB ACTION ALERT:  The Senate Appropriations Committee is expected to consider the Labor, Health and Human Services (LHHS) bill that will provide fiscal year (FY) 2014 funding for the National Institutes of Health (NIH). Although we don’t know when the House Appropriations Committee will vote on NIH funding, the spending limits in the House Budget Resolution could mean another 18 percent cut for biomedical research – below sequestration! NIH needs $32 billion in FY 2014 to prevent further erosion of the nation’s capacity for biomedical research and provide funding for additional grantees.

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Debate on NPR: Is The FDA’s Caution Hazardous To Our Health?

When it comes to approving new medical treatments, the Food and Drug Administration is balancing the need for patient safety against the urgency of making important new available as quickly as possible.

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Rare Disease Patient Sign-on Letter for Essential Health Benefits

Obama Care Implementation:  Sign on to Ensure Rare Diseases Patients have access to needed treatments, physicians and specialists in new Health Insurance Exchanges

Deadline to sign on is COB Friday, May 31st.

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